Exon Skipping

This Exon Skipping Approach was developed by Dr. Glenn Cruse, Principal Investigator and Assistant Professor in the Department of Molecular Biomedical Sciences at the NC State College of Veterinary Medicine. During Dr. Cruse’s research, a new approach for the technique of antisense oligonucleotide-mediated exon skipping to specifically target and down-regulate IgE receptor expression in mast cells was identified. These findings set a breakthrough for allergic diseases as they are driven by the activation of mast cells and the release of mediators in response to IgE-directed antigens.

The high-affinity IgE receptor (FcεRI) plays a central role in the initiation of allergic responses. The research project looks to target novel genes, which are critical for surface IgE receptor expression. The project will utilize splice-switching oligonucleotides (SSOs) to force expression of a truncated isoform of the target genes to reduce expression of FcεRI in mouse asthma models.

Current Preclinical Study:
Hoth has started a preclinical study for the treatment of asthma and allergic inflammation in collaboration with North Carolina State University (NC State). The study has begun the delivery and distribution of nebulized particles which will enable the therapeutic oligonucleotide (oligo), short DNA and RNA molecules that have a wide range of applications in gene testing. Hoth has appointed Dr. Glenn Cruse to its Scientific Advisory Board and he will oversee the Company’s gene therapy program’s advancements.


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